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Gene Therapy Trial Report

Summary

VY-AADC02 for Parkinson's Disease With Motor Fluctuations (RESTORE-1)


NCTID NCT03562494 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Parkinson's Disease
Disease Ontology Term DOID:14330
Compound Name VY-AADC01
Compound Description AAV-hAADC-2
Sponsor Neurocrine Biosciences
Funder Type Industry
Recruitment Status
Completed
Enrollment Count 14 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant DDC
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intraparenchymal (striatum)
Drug Product Type Viral vector
Target Tissue/Cell Striatum
Delivery System Viral transduction
Vector Type AAV2
Dose 1 9E10 vg/200ul (n=5)
Dose 2 3E11 vg/200ul (n=5)
Dose 3 3.6E12 vg

Study Record Dates


Current Stage Phase1
Submit Date 2018-05-16
Completion Date 2024-10-30
Last Update 2024-12-10

Participation Criteria


Eligible Age 40 Years - 75 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Key Inclusion Criteria: 1. Males and females, 40 to 75 years of age (inclusive) 2. Diagnosis of PD, consistent with United Kingdom Brain Bank Criteria 3. Motor responsiveness to dopaminergic therapy, demonstrated by improvement in Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS III score) 4. Disease duration from diagnosis of ≥4 years 5. An average of ≥3 hours of OFF time (that is, periods of insufficient control of motor PD symptoms) per day over 3 consecutive days as confirmed by the PD Diary 6. A stable, optimal regimen of Parkinson's medications including levodopa for at least weeks prior to screening evaluation. Participants must have a minimum duration of levodopa treatment of ≥1 year 7. In the judgment of the Investigator, stable Parkinson's features and symptoms for at least 4 weeks prior to screening evaluation 8. Agrees to defer any elective neurological surgery, including deep brain stimulation or ablation procedure for PD, levodopa or apomorphine infusion, or the addition of new dopaminergic formulations until after the study is completed, if medically appropriate 9. Ability to travel to study visits Key Exclusion Criteria: 1. Atypical or secondary parkinsonism, including but not limited to symptoms believed to be due to trauma, brain tumor, infection, cerebrovascular disease, other neurological disease, or to drugs, chemicals, or toxins, as determined by the Investigator 2. Montreal Cognitive Assessment (MoCA) score \<26 3. New or unstable psychiatric conditions (psychosis, depression) within 1 year of screening 4. Brain imaging abnormalities in the striatum or other regions that would substantially increase risk of surgery 5. Contraindication to magnetic resonance imaging (MRI) and/or gadolinium-based contrast agents 6. Prior brain surgery, infusion therapies or planned treatments that could complicate the study procedure or negatively impact study evaluations as determined from participant interview, screening MRI, or medical records 7. History of malignancy other than treated carcinoma in situ within 3 years of screening evaluation 8. Prior gene transfer, current treatment with any investigational agent (drug or device) within 2 months of screening evaluation, or participation or plans to participate in another research study 9. Severe, biphasic and/or uncontrolled dyskinesia 10. Disabling or uncontrolled impulse control disorders
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 11
Locations United States

Regulatory Information


Has US IND True
FDA Designations Regenerative Medicine Advanced Therapy
Recent Updates Avigen transferred AAV-based product rights to Genzyme Corporation in December 2005, Genzyme launched a collaboration with Voyager Therapeutics in February 2015, then this collaboration was dissolved by Sanofi-Genzyme in October 2017, Voyager entered a collaborative agreement with Neurocrine Biosciences Inc. in March 2019, Neurocrine terminated this agreement effective August 2021, Voyager announced they were terminating this program in 2022

Resources/Links